Red Blood Cell Survival in Sickle Cell Disease

Description

This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion therapy (CTT). Chronic transfusion in patients with SCD is a common treatment. The efficacy of RBC transfusion therapy to treat or prevent complications of SCD may be hampered by variable survival of the transfused donor RBC. The overall aim is to see how long RBC survive in SCD patients who are chronically transfused. When a study participant has a regular blood transfusion the researchers will label a small portion of the RBCs that are transfused with biotin. The participant will return at Day 1, weekly for 3 months and monthly for 3 months to measure how long those RBCs survive. An optional sub-study using INTERCEPT RBCs will mirror the main study but will use INTERCEPT RBCs that have biotinylated for 1 RBC unit.

Conditions

Sickle Cell Disease

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Study Overview

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Study Details

Study overview

This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion therapy (CTT). Chronic transfusion in patients with SCD is a common treatment. The efficacy of RBC transfusion therapy to treat or prevent complications of SCD may be hampered by variable survival of the transfused donor RBC. The overall aim is to see how long RBC survive in SCD patients who are chronically transfused. When a study participant has a regular blood transfusion the researchers will label a small portion of the RBCs that are transfused with biotin. The participant will return at Day 1, weekly for 3 months and monthly for 3 months to measure how long those RBCs survive. An optional sub-study using INTERCEPT RBCs will mirror the main study but will use INTERCEPT RBCs that have biotinylated for 1 RBC unit.

Kinetics of Donor Red Blood Cell Survival in Sickle Cell Disease

Red Blood Cell Survival in Sickle Cell Disease

Condition
Sickle Cell Disease
Intervention / Treatment

-

Contacts and Locations

Atlanta

Hughes Spalding Children's Hospital, Atlanta, Georgia, United States, 30303

Atlanta

Childrens Healthcare of Atlanta, Atlanta, Georgia, United States, 30322

Atlanta

Grady Health System, Atlanta, Georgia, United States, 30322

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Hemoglobinopathy:
  • * Any sickle cell disease genotype, or
  • * Transfusion-dependent thalassemia (TDT)
  • * Receiving CTT for ≥3 months prior to enrollment.
  • * For participants with past BioRBC transfusion exposure, BioRBC antibody screens must have been conducted through at least 6 months post exposure, with negative results.
  • * Anticipated cessation of CTT in the next ≤2 months
  • * Ongoing consumption of biotin or raw egg dietary supplements
  • * Antibody specific of INTERCEPT RBCs at baseline (for subjects consenting to the optional arm)
  • * BioRBC-specific antibodies ever detected in the past, or detected on post-enrollment screening prior to first infusion of Bio-RBC.

Ages Eligible for Study

2 Years to 65 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Emory University,

Marianne Yee, MD, PRINCIPAL_INVESTIGATOR, Emory University

Study Record Dates

2025-05